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Original Article
Allogeneic Hematopoietic Stem Cell Transplantation in Severe Aplastic Anemia: A Single Centre Experience in Malaysia
Gilbert Wilfred1,2, Tee Chuan Ong2, Syed Abd Kadir SH Shahnaz2, Ho Kim Wah2, Edmund Syed Carlo3, Sathar Jameela2, Sen Mui Tan2

1Department of Haematology, Hospital Queen Elizabeth, Ministry of Health Malaysia Sabah

2Department of Haematology, Hospital Ampang, Ministry of Health Malaysia Selangor

3Centre of Clinical Research, Hospital Ampang, Ministry of Health Malaysia Selangor

Keywords
haematopoietic stem cell transplant, severe aplastic anemia, graft failure, graft versus host disease
Submitted:September 2, 2021
Accepted:November 28, 2021
Published online:April 8, 2022

Abstract

Background: Hematopoietic stem cell transplantation (HSCT) provides curative therapy in almost 90% of patients with severe aplastic anemia (SAA). Older age, long duration of disease with consequent heavy exposure to transfusion, and active infection at the time of HSCT have a negative influence on the outcomes, causing graft failure (GF) and graft versus host disease (GVHD).

Purpose: To describe the outcomes of all patients with SAA who received hematopoietic stem cell transplantation at a tertiary center in Malaysia.

Materials and methods: We included a 20 y cohort of patients who underwent transplantation from January 1, 1999 to December 31, 2019. Data were obtained from electronic medical records. Demographics, clinical characteristics, and treatment outcomes were analyzed using descriptive statistics. Overall survival (OS) was analyzed using Kaplan-Meier curves. All analyses were conducted using the Statistical Package for the Social Sciences (SPSS) version 25.

Results: Eighty patients were analyzed. The median age at diagnosis was 19 years, and 59% patients were male (n = 47). Malay ethnicity was the highest (52.5%), followed by Chinese (20.0%) and Native Sabah (15.0%). The median duration from diagnosis to transplantation was 13.5 weeks. A majority of patients received Cy-ATG conditioning (n = 51, 63.8%). Forty-one patients (51.2%) used peripheral blood as stem cell source, 36 patients (45.0%) used granulocyte colony stimulating factor (G-CSF) primed marrow graft and 3 patients (3.8%) used both. The mean nucleated mononuclear cell and CD34 cell doses were 4.7 ± 1.7 × 108/kg and 4.6 ± 1.9 × 106/kg, respectively. Median engraftment for WBCs and platelets was 14 and 15 days, respectively. There was no difference in WBC and platelet engraftment in patients who received peripheral blood stem cell transplantation or bone marrow transplant. At a median follow-up of 54 months, 49 patients (61.3%) achieved complete remission and 8 patients (10.0%) achieved partial remission. The estimated 5 y OS was 63% and higher among those who received HSCT within 3 months of diagnosis. Twenty-two patients (27.5%) died within 100 d of transplantation, and a majority of these died due to pre-engraftment death.

Discussion and conclusions: Our study found that patients who received early allogeneic transplantation for SAA had better outcomes. Pre-engraftment failure was the major cause of transplant-related mortality within 100 d. Further studies are required to identify the factors responsible for delaying transplantation to improve treatment outcomes.

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Online ISSN:2432-7026